Genetic testing could unlock new treatments for kids with brain cancer

NCT ID NCT04859543

Knowledge-focused Sponsor: Masaryk University Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tested whether a detailed genetic analysis of tumor tissue could find treatment targets in 80 children with recurrent or very high-risk brain tumors. The goal was to see if this approach is practical and can help doctors choose personalized therapies. No new drugs were given; the study focused on gathering information.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

What this could lead to: If successful, this could show that genetic testing helps doctors choose better treatments for children with aggressive brain tumors.
What could go wrong: This was a small, early study that only looked at feasibility, not whether the treatments actually work. The results may not apply to all patients.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Not a phased trial

Phase numbers describe drug development. The registry uses this when they do not apply, as it does for trials of devices, procedures or behaviour changes, and for observational studies.

Participants

80 people

The number who actually took part.

Started

Feb 2021

Finished

Dec 2024

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

1 year to 19 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Patient Informed consent form must be appropriately obtained under the applicable local and regulatory requirements. Each patient must sign a consent form prior the enrollment to document their willingness to participate. 2. The subject is male or female, aged 1 - 19 years 3. The subject must have a histologically proven recurrent/ refractory or very high-risk CNS tumors 4. Patients must be in good overall physical condition, which allows tumor biopsy 5. Patients must have a life expectancy of at least 3 months. 6. Patients must have a tumor amenable to image-guided or direct vision biopsy and be willing and able to undergo a tumor biopsy and/or blood taking for molecular profiling. 7. Patients must be accessible for follow-up. Exclusion Criteria: 1. Patients with known psychiatric or substance abuse disorders that would interfere with cooperation with the requirements of the trial. 2. Pregnant and/or breastfeeding women, if applicable 3. No intention to treat the patient.

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Conditions

The condition(s) this trial relates to.

brain cancer brain neoplasm central nervous system cancer central nervous system neoplasm childhood malignant neoplasm neoplasm

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Central nervous system neoplasms GEN1 gene mutation Pediatric cancer Tumor, brain

Genetic testing could unlock new treatments for kids with brain cancer

What the study statuses mean